UniQure Stock Drops After FDA Rejects Rare Disease Gene Therapy
UniQure’s shares plunged after an FDA advisory committee officially rejected its gene therapy for a rare metabolic disease and FDA’s Marty Makary publicly criticized the drug’s clinical data. A class action suit was filed for investors who bought shares between September 24 and October 31, 2025, seeking recovery of losses.
1. FDA Rejection of Gene Therapy
UniQure submitted an experimental gene therapy targeting a rare metabolic disorder to the FDA, but the advisory committee voted against approval, citing insufficient efficacy evidence and unresolved safety issues.
2. Public Criticism by Marty Makary
FDA’s head of research communications, Marty Makary, publicly condemned the clinical trial data as flawed and urged regulators to withhold approval based on the limited patient benefit demonstrated.
3. Stock Market Reaction
Following the rejection and public rebuke, UniQure shares tumbled sharply, wiping out over a quarter of market value in a single trading session and raising investor concerns about its development pipeline.
4. Class Action Lawsuit
A securities class action was filed on behalf of investors who acquired shares between September 24 and October 31, 2025, alleging misleading statements regarding the gene therapy’s approval prospects and clinical robustness.